Ignite Creation Date:
2024-05-05 @ 11:28 AM
Last Modification Date:
2024-10-26 @ 9:08 AM
Study NCT ID:
NCT00059280
Status:
COMPLETED
Last Update Posted:
2014-02-05
First Post:
2003-04-22
Brief Title:
A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
An Open-label Multicenter Multinational Study of the Safety Efficacy Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease
Status:
COMPLETED
Status Verified Date:
2006-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Pompe disease also known as glycogen storage disease type II GSD-II is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase GAA Normally GAA is used by the bodys cells to break down glycogen a stored form of sugar within specialized structures called lysosomes In patients with Pompe disease an excessive amount of glycogen accumulates and is stored in various tissues especially heart and skeletal muscle which prevents their normal function This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase rhGAA as a potential enzyme replacement therapy for Pompe disease Patients diagnosed with infantile-onset Pompe disease who are less than or equal to 6 months old will be studied
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None